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Konferans Briefings

Swiv sou yon Novel Adeno-Associated Virus (AAV) Terapi Jèn (FLT180a) Reyalize Nivo Nòmal Aktivite FIX nan Pasyan Emofili B grav (HB) (Etid B-AMAZE)

Pratima Chowdary, MRCP, FRCPath

Premye-an-imen Etid Terapi Gene nan Teknoloji vektè kapsid AAVhu37 nan emofili grav A - BAY 2599023 gen elijibilite pasyan laj ak ki estab ak soutni alontèm ekspresyon de FVIII

Steven W. Pipe, MD

Karakterizasyon Adeno-Associated Persistence Vektè Viris Apre Long-Term Swivi nan Emofili a Yon Modèl chen

Pòl Batty, MBBS, PhD

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), yon vektè amelyore pou transfè jèn nan granmoun ki gen emofil grav oswa modere-grav: de ane done ki sòti nan yon faz 2b esè.

Steven W. Pipe, MD 

Efikasite ak Sekirite nan Etranacogene Dezaparvovec nan Adilt ki gen grav oswa modere-grav Emofili B: Premye done ki soti nan faz 3 Hope-B Jij Terapi Jijman an

Steven W. Pipe, MD

Premye done ki soti nan faz 3 HOPE-B Jijman Terapi Jij la: efikasite ak sekirite nan Etranacogene Dezaparvovec (AAV5-Padova hFIX Variant; AMT-061) nan granmoun ki gen grav oswa modere-grav Emofili B trete Irrespective of Pre-ki deja egziste anti-kapsid netralizant

Steven W. Pipe, MD

Etranacogene Dezaparvovec (AAV5-Padua hFIX variant), yon vektè amelyore pou transfè jèn nan granmoun ki gen emofil grav oswa modere-grav

Annette von Drygalski, MD, PharmD

AMT-060 Terapi jèn nan adilt ki gen emofili grav oswa modere-grav B konfime ki eksprime ekspresyon fiks ak rediksyon dirab nan senyen ak faktè IX konsomasyon pou jiska 5 an

Wolfgang A. Miesbach, MD

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Enfòmasyon Sou

Sipòte pa sibvansyon edikasyon bay Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, ak uniQure, Inc.

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